Becker's Muscular Dystrophy (BMD)
Conditions
Keywords
Becker's Muscular dystrophy, BMD, Metformin, L-citrulline
Brief summary
The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).
Detailed description
This is a single center, not-randomized open study. The study medication consists of L-citrulline and metformin administered orally; 5g citrulline and 500mg metformin tablets will be given 3 times daily. Half of the patients will be treated with metformin during the first 6 weeks of the study. The other patients will receive initially L-citrulline for 6 weeks, before all patients will be treated with the combination therapy (metformin and L-citrulline) for another 6 week period. The study lasts 12 weeks and includes the screening visit and 3 further visits at baseline, week 6 and week 12.
Interventions
DRUGMetformin and Metformin & L-Citrulline
DRUGL-Citrulline and Metformin & L-Citrulline
Sponsors
University Hospital, Basel, Switzerland
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 18 years or older * Molecular or immunohistochemical diagnosis of BMD * ambulant at the time point of screening
Exclusion criteria
* Participation in another therapeutic BMD study within the last 3 months * Use of L-Arginine, L-Citrulline or Metformin within the last 3 months * Other chronic disease or relevant limitation of renal, liver, heart function according to discretion of investigator * known hypersensitivity to L-citrulline or metformin
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks | week 6 and week 12 |
Secondary
| Measure | Time frame |
|---|---|
| MFM total score and six minute walking distance (6MWD) | week 6 and week 12 |
| Change of muscle fat content (MFC) (assessed by MRI) | week 6 and week 12 |
| Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) | week 6 and week 12 |
| Change of laboratory parameters (oxidative and nitrosative stress) | week 6 and week 12 |
Countries
Switzerland
Outcome results
None listed